~ AMT-130 granted Breakthrough Therapy designation by FDA ~ ~ Initial safety data from third cohort of Phase I/II study show AMT-130 continues to be generally well-tolerated, with no treatment-related serious adverse events ~ ~ Held Type B FDA meetings in the first and second quarters of 2025 to advance BLA preparations for AMT-130; regulatory update expected in the second quarter of 2025 ~ ~ AMT-260 clinical data from first patient to be presented at Epilepsy Therapies & Diagnostics Development Symposium o ...

~ uniQure to host inaugural earnings call on Friday, May 9, 2025 at 8:30 a.m. ET ~LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 05, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs will report first quarter 2025 financial results before market open on Friday, May 9, 2025. Management will then host a conference call at 8:30 a.m. ET. As the Company advances its pipeline and AMT-130 toward a BLA ...

Company Overview - uniQure (QURE) shares increased by 38.5% in the last trading session, closing at $13, following a notable volume of shares traded, contrasting with a 33.1% loss over the past four weeks [1] - The stock's rally was driven by the FDA granting Breakthrough Therapy designation to its pipeline candidate, AMT-130, for treating Huntington's disease, a rare neurodegenerative disorder [1] Financial Performance - The company is expected to report a quarterly loss of $1.07 per share, reflecting a year-over-year increase of 21.3%, while revenues are projected to be $6 million, down 29.3% from the same quarter last year [2] - The consensus EPS estimate for uniQure has remained unchanged over the last 30 days, indicating that stock price movements may not sustain without trends in earnings estimate revisions [3] Industry Context - uniQure is part of the Zacks Medical - Biomedical and Genetics industry, where Kiniksa Pharmaceuticals, Ltd. (KNSA) experienced a 1.6% decline in the last trading session, with a -11% return over the past month [3] - Kiniksa Pharmaceuticals has a consensus EPS estimate of $0.06 for its upcoming report, representing a 124% increase from the previous year, and currently holds a Zacks Rank of 2 (Buy) [4]

Core Insights - The FDA has granted Breakthrough Therapy designation to AMT-130 for Huntington's disease, highlighting the urgent need for effective treatments and the promising interim data indicating potential disease progression slowing [1][2] - The designation is supported by clinical data from ongoing Phase I/II trials, with interim data showing dose-dependent slowing of disease progression based on the cUHDRS after 24 months [2] Regulatory Updates - Additional regulatory updates and guidance on the Biologics License Application (BLA) submission for AMT-130 are expected in the second quarter of 2025 [1] - The Breakthrough Therapy designation allows for expedited development and review processes, including intensive guidance on drug development and senior management involvement from the FDA [3] Huntington's Disease Overview - Huntington's disease is a rare, inherited neurodegenerative disorder affecting approximately 70,000 diagnosed individuals in the U.S. and Europe, with no approved therapies to slow its progression [4] - The disease is characterized by motor symptoms, behavioral abnormalities, and cognitive decline due to a genetic mutation in the huntingtin gene [4] Company Background - uniQure is focused on advancing gene therapies, including AMT-130 for Huntington's disease, and has previously achieved significant milestones with its gene therapy for hemophilia B [5] - The company is developing a pipeline of gene therapies for various severe diseases, including ALS and Fabry disease [5]

~ Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~ ~ Additional regulatory update and guidance on the Biologics License Application submission expected in the second quarter of 2025 ~ LEXINGTON, Mass. and AMSTERDAM, April 17, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and ...

UniQure (QURE) came out with a quarterly loss of $1.50 per share versus the Zacks Consensus Estimate of a loss of $0.44. This compares to loss of $1.53 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -240.91%. A quarter ago, it was expected that this human gene therapy company would post a loss of $1.12 per share when it actually produced a loss of $0.91, delivering a surprise of 18.75%.Over the last four quarters, the company ...

Financial Performance - The company reported net losses for the years ended December 31, 2024 and 2023, with significant losses in previous years and expects to incur losses in the future[16] - The company reported net losses for the years ended December 31, 2024 and 2023, and anticipates continued losses in the future, indicating a risk of never achieving profitability[16] - The company has incurred significant losses in previous years and will need to raise additional funding to advance product development, which may not be available on acceptable terms[18] Product Development and Clinical Trials - The lead product candidate, AMT-130, is critical for the treatment of Huntington's disease, and any failure in its clinical development could adversely affect the company's business[15] - The company may face delays in clinical trials and challenges in demonstrating the safety and efficacy of its product candidates, which could impact future operations[15] - The company may face delays in clinical trials and challenges in demonstrating safety and efficacy of its product candidates, which could materially harm its business[15] - The company has encountered risks associated with the marketing approval process for gene therapies, which may affect the commercialization of its product candidates[15] Regulatory and Manufacturing Risks - The company has encountered risks related to the manufacturing of gene therapies, which are complex and expensive, potentially leading to delays in development or commercialization[15] - The company is dependent on third-party manufacturers for its product candidates, which may not perform satisfactorily or comply with regulatory requirements[15] - The company relies on third parties for manufacturing its product candidates, which may not perform satisfactorily or comply with regulations[15] - The company plans to leverage specialized regulatory pathways, such as the FDA's accelerated approval pathway, but may struggle to maintain the benefits associated with these designations[15] - The company may leverage specialized regulatory pathways for its product candidates, but there is no guarantee of obtaining or maintaining the associated benefits[15] Market and Competitive Landscape - The market price of the company's ordinary shares has been volatile and may fluctuate substantially in the future[18] - The company faces substantial competition in the industry, which may impact its ability to successfully commercialize its products[18] - The company faces substantial competition and risks that competitors may develop or commercialize competing products more successfully[18] Business Development and Strategic Initiatives - The company’s business development initiatives, including acquisitions and partnerships, may not achieve their intended benefits, posing additional risks[15] - The Lexington Transaction may not yield expected benefits and could introduce additional risks related to manufacturing and supply requirements[15]

Financial Performance - Revenue for the year ended December 31, 2024, was $27.1 million, an increase of 71.4% compared to $15.8 million in 2023[6] - Total revenues for 2024 reached $27,119,000, a significant increase from $15,843,000 in 2023, representing a growth of 71.5%[22] - License revenues increased to $10,133,000 in 2024, compared to $2,758,000 in 2023, marking a growth of 267.5%[22] - Collaboration revenues rose to $10,872,000 in 2024, up from $2,250,000 in 2023, reflecting a growth of 384.4%[22] - The net loss for the year ended December 31, 2024, was $239.6 million, a decrease from a net loss of $308.5 million in 2023[14] - Net loss for 2024 was $239,556,000, an improvement from a net loss of $308,478,000 in 2023, indicating a reduction of 22.4%[22] - Basic and diluted net loss per ordinary share improved to $4.92 in 2024 from $6.47 in 2023[22] Expenses - Research and development expenses decreased to $143.8 million in 2024 from $214.9 million in 2023, reflecting a reduction of $71.1 million[9] - Selling, general and administrative expenses were $52.7 million for the year ended December 31, 2024, down from $74.6 million in 2023, a decrease of $21.9 million[10] - Total operating expenses decreased to $214,766,000 in 2024 from $303,083,000 in 2023, a reduction of 29.1%[22] Cash and Assets - Cash and cash equivalents as of December 31, 2024, were approximately $367.5 million, with proforma cash including net proceeds from a recent offering totaling around $448 million[5] - Total current assets decreased to $390,289,000 in 2024 from $651,853,000 in 2023, a decline of 40.1%[20] - Cash and cash equivalents decreased to $158,930,000 in 2024 from $241,360,000 in 2023, a decline of 34.2%[20] Liabilities and Equity - Total liabilities decreased to $563,288,000 in 2024 from $624,019,000 in 2023, a reduction of 9.7%[20] - Total shareholders' equity turned negative at $(6,752,000) in 2024, down from $207,670,000 in 2023[20] Clinical Developments - The company completed enrollment of all 12 patients in the third cohort of the AMT-130 study in February 2025, with an initial safety update expected in Q2 2025[4] - The FDA granted RMAT designation for AMT-130 in May 2024, indicating its potential to address unmet medical needs for Huntington's disease[4] - The company expects to fund operations into the second half of 2027 based on its current cash position and planned U.S. launch of AMT-130[5] - The company initiated dosing of the Phase I/II study of AMT-260 in mesial temporal lobe epilepsy, with protocol changes to expand inclusion criteria[2] - The company anticipates presenting data from ongoing Phase I/II studies of AMT-130 in support of a potential BLA submission in Q3 2025[4]

Core Insights - uniQure has made significant clinical and operational progress in 2024, particularly with AMT-130 for Huntington's disease, including alignment with the FDA on the Accelerated Approval pathway [2][5] - The company has completed patient enrollment in various Phase I/II studies, including AMT-130, AMT-260, AMT-191, and AMT-162, and has received favorable recommendations from Independent Data Monitoring Committees [1][2] - Financially, uniQure reported a cash position of approximately $367.5 million as of December 31, 2024, with net proceeds from a recent financing expected to fund operations into the second half of 2027 [1][7] Clinical Developments - The FDA granted RMAT designation for AMT-130, indicating its potential to address unmet medical needs in Huntington's disease [2] - The company has initiated preparations for a Biologics License Application (BLA) submission for AMT-130, with the FDA agreeing that ongoing Phase I/II study data may serve as the primary basis for this submission [2][5] - Enrollment in the Phase I/II studies of AMT-191 for Fabry disease and AMT-162 for SOD1-ALS is progressing, with the second cohorts expected to begin dosing soon [1][2] Financial Performance - Revenue for the year ended December 31, 2024, was $27.1 million, an increase from $15.8 million in 2023, driven by higher license and collaboration revenues [8][20] - Research and development expenses decreased to $143.8 million in 2024 from $214.9 million in 2023, reflecting cost-cutting measures [10] - Selling, general, and administrative expenses also decreased to $52.7 million in 2024 from $74.6 million in 2023, indicating improved operational efficiency [11] Strategic Actions - The company completed the sale of its Lexington manufacturing facility and underwent a restructuring that reduced its workforce by approximately 65%, significantly lowering cash burn by about $70 million per year [2][7] - A public offering of 5.1 million ordinary shares at $17.00 per share was completed, providing additional funding for operations [7][8] - Upcoming investor events are scheduled, including participation in healthcare conferences in March and April 2025 [4][6]

Core Insights - The Independent Data Monitoring Committee (IDMC) has recommended proceeding with the enrollment of the second cohort in the Phase I/IIa trial of AMT-191 after reviewing safety data from the first cohort [1][2] - The company expects to initiate enrollment of the second dose cohort in the first quarter of 2025 [1] Company Overview - uniQure N.V. is a leading gene therapy company focused on developing transformative therapies for patients with severe medical needs [1][6] - The company has a pipeline of proprietary gene therapies targeting various severe diseases, including Huntington's disease, refractory temporal lobe epilepsy, ALS, and Fabry disease [6] Clinical Trial Details - The Phase I/IIa clinical trial of AMT-191 is a multi-center, open-label study in the U.S. involving two dosing cohorts of up to six adult male patients each [3] - Patients will receive intravenous infusions of AMT-191 while continuing their regular enzyme replacement therapy for a period of 24 months [3] - The trial aims to explore safety, tolerability, and early signs of efficacy by measuring the expression of the lysosomal enzyme α-galactosidase A (aGLA-A) [3] Disease Context - Fabry disease is an X-linked genetic disorder caused by a deficiency of the GLA enzyme, leading to lipid accumulation in various cell types, including kidney and heart cells [2][5] - The prevalence of Fabry disease is estimated to be between one in 40,000 and one in 117,000 individuals [5] - Current standard care involves bi-weekly infusions of enzyme replacement therapy, which has limited effectiveness due to poor cross-correction [5] Regulatory Status - AMT-191 has received Orphan Drug status and Fast Track designation from the U.S. Food and Drug Administration [4]